New Medicines Available To Treat Rare Diseases
Below is a MRR and PLR article in category Health Fitness -> subcategory Medicine.
New Medicines Bring Hope for Rare Disease Patients
Since 1995, America's pharmaceutical research companies have achieved remarkable progress in the fight against rare diseases. Over the past decade, the Food and Drug Administration (FDA) has approved more than 160 new medicines designed to treat these rare or "orphan" diseases.
The National Institutes of Health reports that over 6,000 rare diseases affect 25 million Americans. Alarmingly, the FDA indicates that 85 to 90 percent of these diseases are serious or life-threatening, underscoring the critical need for new treatments and cures.
"America's pharmaceutical research companies have made incredible progress in finding new medicines that offer hope to patients with rare diseases," says Billy Tauzin, president and CEO of the Pharmaceutical Research and Manufacturers of America (PhRMA). "Ongoing innovation by these companies is crucial for developing new medicines and providing patients with a brighter, healthier future."
The report "A Decade of Innovation: Advances in the Treatment of Rare Diseases," issued by the National Organization for Rare Disorders, the Genetic Alliance, and PhRMA, highlights significant drug approvals by the FDA over the past decade. These breakthroughs have transformed patients' lives. For example:
- Rilutek (riluzole): The first drug to demonstrate increased survival in patients with amyotrophic lateral sclerosis (ALS) or Lou Gehrig's disease. ALS is a progressive disease affecting motor nerve cells in the brain and spinal cord, ultimately leading to loss of muscle control.
- Fabrazyme (agalsidase beta): The first medication to address the root cause of Fabry disease. This condition, primarily affecting men, results from a deficiency in an enzyme needed to break down fats, causing harmful buildups in blood vessels and organs. While patients often survive into adulthood, they face increased risks of stroke, heart attack, and heart disease.
While researchers have made notable strides, efforts continue to develop new treatments for individuals with rare conditions. Ongoing research targets diseases like Crohn's disease, chronic myeloid leukemia, pulmonary arterial hypertension, and many other rare disorders.
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